Observational Study to Characterize Real-world Clinical Outcomes With Relapsing-remitting Multiple Sclerosis (RRMS) (EFFECT)

A Multicenter, Global, Retrospective, Observational Study to Characterize Real-world Clinical Outcomes in Patients With Relapsing-remitting Multiple Sclerosis Treated With Disease-modifying Therapies (Tecfidera®, Copaxone®, Aubagio®, or Gilenya®)

The primary objective of the study is to evaluate the real-world clinical effectiveness, as measured by the proportion of participants relapsed at 12 months, in participants treated with dimethyl fumarate (DMF).

Secondary objectives of the study are: To evaluate the real-world clinical effectiveness, as measured by the proportion of participants relapsed at 12 months, in participants treated with DMF, glatiramer acetate (GA), teriflunomide, or fingolimod both in the overall participant cohort and in a subset of participants who were naïve to disease-modifying therapy (DMT) and were diagnosed with multiple sclerosis (MS) within 3 years of starting the index therapy; To compare relapse activity, defined as annualized relapse rate (ARR), among participants treated with DMF, GA, teriflunomide, or fingolimod; To compare MS-related hospitalizations among participants treated with DMF, GA, teriflunomide, or fingolimod; To compare intravenous corticosteroid use among participants treated with DMF, GA, teriflunomide, or fingolimod.

Study Overview

• Status: Completed
• Conditions: Multiple Sclerosis

• Study Type: Observational
• Enrollment (Actual): 2978

Contacts and Locations

Study Locations

• Argentina
  • Ciudad Autonoma Buenos Aires, Argentina, C1428AQK
    • Research Site
  • Cordoba, Argentina, X5000JHQ
    • Research Site
  • Salta, Argentina, 4400
    • Research Site
  • Buenos Aires
    • Ciudad Autonoma de Buenos Aires, Buenos Aires, Argentina, C1061ABD
      • Research Site
    • Ciudad Autonoma de Buenos Aires, Buenos Aires, Argentina, C1425DKT
      • Research Site
  • Santa Fe
    • Rosario, Santa Fe, Argentina, 2000
      • Research Site
    • Rosario, Santa Fe, Argentina, S20000
      • Research Site
• Australia
  • New South Wales
    • New Lambton Heights, New South Wales, Australia, 2305
      • Research Site
  • Victoria
    • Box Hill, Victoria, Australia, 3128
      • Research Site
    • Heidelberg, Victoria, Australia, 3084
      • Research Site
    • Melbourne, Victoria, Australia, 3050
      • Research Site
• Canada
  • British Columbia
    • Burnaby, British Columbia, Canada, V5H 4K7
      • Research Site
  • New Brunswick
    • St. John, New Brunswick, Canada, E2L4L2
      • Research Site
  • Nova Scotia
    • Halifax, Nova Scotia, Canada, B3H4K4
      • Research Site
  • Ontario
    • Cambridge, Ontario, Canada, N1R7L6
      • Research Site
    • Guelph, Ontario, Canada, N1H 4J4
      • Research Site
  • Quebec
    • Chicoutimi, Quebec, Canada, G7H 5H6
      • Research Site
    • Gatineau, Quebec, Canada, J9J 0A5
      • Research Site
    • Greenfield Park, Quebec, Canada, J4V 2J2
      • Research Site
• Croatia
  • Osijek, Croatia, 31000
    • Research Site
  • Varazdin, Croatia, 42000
    • Research Site
• Czech Republic
  • Brno, Czech Republic, 656 91
    • Research Site
  • Hradec Kralove, Czech Republic, 500 05
    • Research Site
  • Hradec Kralove, Czech Republic, 50003
    • Research Site
  • Jihlava, Czech Republic, 58633
    • Research Site
  • Olomouc, Czech Republic, 775 20
    • Research Site
  • Ostrava-Poruba, Czech Republic, 70800
    • Research Site
  • Pardubice, Czech Republic, 532 03
    • Research Site
  • Prague 5, Czech Republic, 150 06
    • Research Site
  • Teplice, Czech Republic, 41501
    • Research Site
• France
  • Gironde
    • Libourne Cedex, Gironde, France, 33505
      • Research Site
  • Haute Garonne
    • Toulouse cedex 9, Haute Garonne, France, 31059
      • Research Site
  • Sarthe
    • Le Mans cedex 9, Sarthe, France, 72037
      • Research Site
  • Yvelines
    • Le Chesnay Cedex, Yvelines, France, 78157
      • Research Site
• Germany
  • Berlin, Germany, 10961
    • Research Site
  • Berlin, Germany, 12099
    • Research Site
  • Hamburg, Germany, 20249
    • Research Site
  • Baden Wuerttemberg
    • Freiburg, Baden Wuerttemberg, Germany, 79098
      • Research Site
    • Pforzheim, Baden Wuerttemberg, Germany, 75172
      • Research Site
    • Stuttgart, Baden Wuerttemberg, Germany, 70182
      • Research Site
  • Bayern
    • Bamberg, Bayern, Germany, 96052
      • Research Site
  • Brandenburg
    • Potsdam, Brandenburg, Germany, 14471
      • Research Site
  • Hessen
    • Kassel, Hessen, Germany, 34121
      • Research Site
  • Niedersachsen
    • Oldenburg, Niedersachsen, Germany, 26122
      • Research Site
    • Osnabrueck, Niedersachsen, Germany, 49076
      • Research Site
    • Stade, Niedersachsen, Germany, 21682
      • Research Site
  • Nordrhein Westfalen
    • Bochum, Nordrhein Westfalen, Germany, 44787
      • Research Site
    • Düsseldorf, Nordrhein Westfalen, Germany, 40211
      • Research Site
    • Gelsenkirchen, Nordrhein Westfalen, Germany, 45879
      • Research Site
    • Hagen, Nordrhein Westfalen, Germany, 58095
      • Research Site
    • Siegen, Nordrhein Westfalen, Germany, 57076
      • Research Site
    • Sprockhoevel, Nordrhein Westfalen, Germany, 45549
      • Research Site
  • Rheinland Pfalz
    • Kandel, Rheinland Pfalz, Germany, 76870
      • Research Site
  • Saarland
    • St. Ingbert, Saarland, Germany, 66386
      • Research Site
  • Sachsen
    • Leipzig, Sachsen, Germany, 04229
      • Research Site
  • Schleswig Holstein
    • Kiel, Schleswig Holstein, Germany, 24105
      • Research Site
• Hungary
  • Budapest, Hungary, 1083
    • Research Site
  • Debrecen, Hungary, 4032
    • Research Site
  • Eger, Hungary, 3300
    • Research Site
  • Esztergom, Hungary, 2500
    • Research Site
  • Kecskemet, Hungary, 6000
    • Research Site
  • Nyiregyhaza, Hungary, 4400
    • Research Site
• Italy
  • Cagliari, Italy, 09126
    • Research Site
  • Catania, Italy, 95123
    • Research Site
  • Napoli, Italy, 80131
    • Research Site
  • Napoli, Italy, 80138
    • Research Site
• Spain
  • Alicante, Spain, 03010
    • Research Site
  • Cordoba, Spain, 14011
    • Research Site
  • Madrid, Spain, 28007
    • Research Site
  • Malaga, Spain, 29010
    • Research Site
  • Sevilla, Spain, 41071
    • Research Site
  • Barcelona
    • Badalona, Barcelona, Spain, 08916
      • Research Site
  • La Coruña
    • Santiago de Compostela, La Coruña, Spain, 15706
      • Research Site
  • Pontevedra
    • Vigo, Pontevedra, Spain, 36312
      • Research Site
• Switzerland
  • Lausanne, Switzerland, CH-1011
    • Research Site
  • Lugano, Switzerland, 6903
    • Research Site
• United Kingdom
  • Cornwall
    • Truro, Cornwall, United Kingdom, TR1 3LJ
      • Research Site
  • Devon
    • Exeter, Devon, United Kingdom, EX2 5DW
      • Research Site
  • Essex
    • Chelmsford, Essex, United Kingdom, CM1 7ET
      • Research Site
    • Romford, Essex, United Kingdom, RM7 0AG
      • Research Site
  • Greater London
    • London, Greater London, United Kingdom, SW17 0QT
      • Research Site
  • Strathclyde
    • Glasgow, Strathclyde, United Kingdom, G51 4TF
      • Research Site
  • Surrey
    • Chertsey, Surrey, United Kingdom, KT16 0PZ
      • Research Site
• United States
  • Alabama
    • Homewood, Alabama, United States, 35209
      • Research Site
  • Arizona
    • Gilbert, Arizona, United States, 85234
      • Research Site
  • California
    • Hanford, California, United States, 93230
      • Research Site
    • Modesto, California, United States, 95355
      • Research Site
    • Santa Ana, California, United States, 92704
      • Research Site
  • Colorado
    • Aurora, Colorado, United States, 80045
      • Research Site
  • Connecticut
    • Danbury, Connecticut, United States, 06810
      • Research Site
    • Hartford, Connecticut, United States, 6112
      • Research Site
  • Florida
    • Jacksonville, Florida, United States, 32256
      • Research Site
    • Port Orange, Florida, United States, 32127
      • Research Site
  • Georgia
    • Savannah, Georgia, United States, 31406
      • Research Site
  • Indiana
    • Avon, Indiana, United States, 46123
      • Research Site
    • Franklin, Indiana, United States, 46131
      • Research Site
    • Muncie, Indiana, United States, 47304
      • Research Site
  • Kansas
    • Overland Park, Kansas, United States, 66212
      • Research Site
  • Massachusetts
    • Boston, Massachusetts, United States, 02215
      • Research Site
    • Boston, Massachusetts, United States, 02135
      • Research Site
    • Wellesley, Massachusetts, United States, 02481
      • Research Site
  • Minnesota
    • Golden Valley, Minnesota, United States, 55422
      • Research Site
  • New York
    • Amherst, New York, United States, 14226
      • Research Site
    • New Hyde park, New York, United States, 11042
      • Research Site
    • New York, New York, United States, 10019
      • Research Site
    • Patchogue, New York, United States, 11772
      • Research Site
  • North Carolina
    • Raleigh, North Carolina, United States, 27607-6010
      • Research Site
  • Ohio
    • Cleveland, Ohio, United States, 44195
      • Research Site
    • Columbus, Ohio, United States, 43210
      • Research Site
    • Columbus, Ohio, United States, 43221
      • Research Site
  • Oregon
    • Medford, Oregon, United States, 97504
      • Research Site
    • Portland, Oregon, United States, 97225
      • Research Site
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19107
      • Research Site
    • Pittsburgh, Pennsylvania, United States, 15212
      • Research Site
  • Texas
    • Dallas, Texas, United States, 75246
      • Research Site
    • Houston, Texas, United States, 77018
      • Research Site
    • Mansfield, Texas, United States, 76063
      • Research Site
    • Round Rock, Texas, United States, 78681
      • Research Site
  • Utah
    • Salt Lake City, Utah, United States, 84103
      • Research Site
  • Virginia
    • Norfolk, Virginia, United States, 23502
      • Research Site
  • Washington
    • Tacoma, Washington, United States, 98405
      • Research Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

Participants with a diagnosis of RRMS treated with dimethyl fumarate, fingolimod, teriflunomide, or glatiramer acetate in routine clinical practice.

Description

Key Inclusion Criteria:

• Diagnosis of RRMS per McDonald criteria
• Initiated treatment with DMF, GA, teriflunomide, or fingolimod (defined as index therapy) no earlier than January 2011 and no later than 12 months prior to the date of medical record abstraction (i.e., patient has at least 12 months of data available in the medical record following initiation of index treatment). Note: Patients who have initiated the index therapy but subsequently discontinued or switched to other therapies are allowed in this study as long as there is at least 12 months of follow-up following the initiation of the index therapy.
• Have sufficient available medical records for data abstraction to meet the objectives of the study, i.e., the patient was either under the medical care of the investigating site during the entire period of the index treatment or the patient's complete MS disease and treatment history is otherwise available at the investigating site

Key Exclusion Criteria:

• Diagnosis of a progressive form of MS (progressive relapsing, primary progressive, secondary progressive) at any time before or during the period for which data will be collected
• Have received disease-modifying therapies other than one platform therapy (IFN or GA) prior to initiation of index therapy. In patients for whom GA is index therapy, only one prior IFN therapy is allowed.
• Have received any formulations of DMF, Fumaderm®, or compounded fumarates (e.g., Psorinovo) at any time prior to initiation of treatment with Tecfidera®
• Concurrent enrollment in any interventional clinical trial of an investigational product during time evaluated for medical record abstraction

NOTE: Other protocol defined Inclusion/Exclusion criteria may apply

Study Plan

How is the study designed?

Number of groups / cohorts

4

Cohorts and Interventions

Group / Cohort
dimethyl fumarate (DMF); Participants who initiated DMF during the specified time period
glatiramer acetate (GA); Participants who initiated GA during the specified time period
teriflunomide; Participants who initiated teriflunomide during the specified time period
fingolimod; Participants who initiated fingolimod during the specified time period

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Proportion of participants relapsed at 12 months in participants treated with DMF	Single time point per participant retrospective medical record abstraction with no required study visits or procedures	One day

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Proportion of participants relapsed at 12 months treated with index therapy in the overall population as well as the subgroup of MS participants who were naïve to DMTs and were diagnosed with MS within 3 years of starting the index therapy	Single time point per participant retrospective medical record abstraction with no required study visits or procedures	One day
ARR at 12 months in participants treated with index therapy in the overall population as well as in the matched cohorts	Single time point per participant retrospective medical record abstraction with no required study visits or procedures	One day
Proportion of relapsed participants with one or more MS-related hospitalizations during the 12 months following treatment initiation in participants treated with index therapy in the overall population and the matched cohorts	Single time point per participant retrospective medical record abstraction with no required study visits or procedures	One day
Proportion of relapsed participants requiring treatment with intravenous corticosteroids during the 12 months following treatment initiation in participants treated with index therapy in the overall participant population and the matched cohorts	Single time point per participant retrospective medical record abstraction with no required study visits or procedures	One day

Collaborators and Investigators

• Sponsor: Biogen

Publications and helpful links

General Publications

• Min J, Cohan S, Alvarez E, Sloane J, Phillips JT, van der Walt A, Koulinska I, Fang F, Miller C, Chan A. Real-World Characterization of Dimethyl Fumarate-Related Gastrointestinal Events in Multiple Sclerosis: Management Strategies to Improve Persistence on Treatment and Patient Outcomes. Neurol Ther. 2019 Jun;8(1):109-119. doi: 10.1007/s40120-019-0127-2. Epub 2019 Jan 31.

Study record dates

Study Major Dates

• Study Start: May 1, 2016
• Primary Completion (Actual): December 1, 2016
• Study Completion (Actual): December 1, 2016

Study Registration Dates

• First Submitted: May 16, 2016
• First Submitted That Met QC Criteria: May 16, 2016
• First Posted (Estimate): May 18, 2016

Study Record Updates

• Last Update Posted (Actual): March 23, 2017
• Last Update Submitted That Met QC Criteria: March 21, 2017
• Last Verified: March 1, 2017

More Information

Terms related to this study

• Keywords: Tecfidera, DMF, dimethyl fumarate, Fingolimod, Glatiramer acetate, Teriflunomide, Gilenya, Copaxone, Aubagio
• Additional Relevant MeSH Terms: Pathologic Processes; Nervous System Diseases; Immune System Diseases; Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Demyelinating Diseases; Autoimmune Diseases; Multiple Sclerosis; Sclerosis; Multiple Sclerosis, Relapsing-Remitting
• Other Study ID Numbers: 109MS421